At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. In many cases, development is being accelerated by our gene therapy engine, which potentially provides a more efficient model for drug design.
Sarepta has two FDA-approved therapies.
Sarepta is at the forefront of precision genetic medicine, with over 40 therapies in various stages of development. Our disease areas include Duchenne muscular dystrophy (DMD), six Limb-girdle muscular dystrophies (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA (Sanfilippo Syndrome type A), and other CNS-related disorders. The company’s programs and research focus span several therapeutic modalities, including gene therapy, RNA technologies, and gene editing.
Sarepta is committed to creating breakthrough treatments for people whose lives are being cut short and burdened by rare diseases. Fundamental to that mission are carefully designed and conducted clinical trials.
Our access programs include the SareptAssist Patient Support Program and Compassionate Use Policy and Managed Access Program (MAP).